Julie Parsons, MD, is a professor of clinical pediatrics and neurology and co-director of the Neuromuscular Clinic at Children’s Hospital Colorado
Final Panel Thoughts on the Management of SMA
June 26th 2025Panelists discuss how spinal muscular atrophy (SMA) treatment will evolve over the next 5 years, likely incorporating improved gene therapy delivery systems, combination therapies, and rehabilitation models, while maintaining individualized approaches for each patient.
Optimizing Multidisciplinary Care Approaches for the Future of SMA Management
June 26th 2025Panelists discuss how communication between clinicians, patients, and payers could improve equitable access to spinal muscular atrophy (SMA) treatments, while acknowledging the complex value assessment of high-cost therapies vs improved quality and length of life.
Insurance Considerations for SMA Therapies
June 19th 2025Panelists discuss how insurance challenges impact spinal muscular atrophy (SMA) care, with prior authorizations becoming more streamlined over time but limitations on physical therapy sessions and inequitable access to treatments remain significant barriers.
Other Investigational Treatment Options for SMA
June 19th 2025Panelists discuss how combination therapies targeting different aspects of spinal muscular atrophy (SMA), such as myostatin inhibitors (targeting muscle) alongside SMN protein-enhancing treatments, hold promise for addressing fatigue and improving muscle function in older patients.
Evaluating the Potential of Intrathecal Gene Therapy
June 12th 2025Panelists discuss how emerging intrathecal gene therapy for older patients with spinal muscular atrophy (SMA) shows modest efficacy with fewer systemic adverse effects than intravenous administration, though safety concerns and limited data remain.
Assessing Different Factors When Selecting a Treatment
June 12th 2025Panelists discuss how comorbidities such as scoliosis, dislocated hips, and nutritional issues affect spinal muscular atrophy (SMA) treatment decisions, with treatment approaches evolving as patients' functional abilities improve with disease-modifying therapies.
Key Clinical Considerations for the Management of SMA
June 5th 2025Panelists discuss how disease progression despite treatment in older patients with spinal muscular atrophy (SMA) may be due to natural aging effects combined with SMA, not necessarily treatment failure, highlighting the importance of exercise, nutrition, and management of contractures.
Splicing Modifiers and Gene Therapy for SMA
June 5th 2025Panelists discuss how splice modifiers work by enhancing protein production from the SMN2 gene, with risdiplam (Evrysdi) being an oral daily medication and nusinersen (Spinraza) being administered intrathecally quarterly, both showing similar safety and efficacy profiles.
Introduction to Therapies Used to Treat SMA
May 29th 2025Panelists discuss how the current spinal muscular atrophy (SMA) treatment landscape includes 3 options: gene therapy (onasemnogene abeparvovec [Zolgensma]) for younger patients and 2 splice modifiers (nusinersen [Spinraza] and risdiplam [Evrysdi]) for older patients.
The Importance of Supportive Care for Patients Living With SMA
May 29th 2025Panelists discuss how supportive care for patients with spinal muscular atrophy (SMA) includes rehabilitation, respiratory care, psychosocial support, and multidisciplinary approaches to help patients achieve independence and improved quality of life.
Describing SMA Progression Over Time
May 15th 2025Panelists discuss how spinal muscular atrophy (SMA) progresses over time, affecting motor function differently in older patients vs infants, with experts noting the shift from traditional classification (types 1 to 4) to functional categories (nonsitters, sitters, walkers).
Key Considerations for Choosing the Right SMA Treatment
April 4th 2025FDA-approved treatments for spinal muscular atrophy differ in their mechanisms of action, safety profiles, and administration challenges, with key selection factors like patient age and gene copy number, according to Julie Parsons, MD, a professor and neurologist from the University of Colorado School of Medicine.